Daily salbutamol in young patients with SMA type II

被引：86

作者：

Pane, Marika ^{[1
]}

Staccioll, Susanna ^{[1
]}

Messina, Sonia ^{[1
,2
]}

D'Arnico, Adele ^{[3
]}

Pelliccioni, Marco ^{[1
,4
]}

Mazzone, Elena S. ^{[1
]}

Cuttini, Marina ^{[5
]}

Alfieri, Paolo ^{[1
]}

Battinif, Roberta ^{[6
,7
]}

Main, Marion ^{[8
]}

Muntoni, Francesco ^{[8
]}

Bertini, Enrico ^{[3
]}

Villanova, Marcello ^{[9
]}

Mercuri, Eugenio ^{[1
]}

机构：

[1] Univ Cattolica Sacro Cuore, Dept Paediat Neurol, I-00168 Rome, Italy

[2] Univ Messina, Dept Neurosci Psychiat & Anaesthesiol, I-98100 Messina, Italy

[3] Bambino Gesu Pediat Hosp, Mol Med Unit, Dept Lab Med, Rome, Italy

[4] Unione Italiana Lotta Distrofia Muscolare, UILDM, Rome, Italy

[5] Bambino Gesu Pediat Hosp, Epidemiol Unit, Rome, Italy

[6] Univ Pisa, Unit Child Neurol & Psychiat, I-56100 Pisa, Italy

[7] IRCCS, San Giovanni Rotondo, Italy

[8] UCL, Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

[9] Nigrisoli Hosp, Bologna, Italy

来源：

NEUROMUSCULAR DISORDERS | 2008年 / 18卷 / 07期

基金：

英国医学研究理事会;

关键词：

spinal muscular atrophy; salbutamol; therapy; children; beta; 2; agonists;

D O I：

10.1016/j.nmd.2008.05.004

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

The aim of this open pilot study was to establish the profile of tolerability and clinical response of salbutamol (albuterol) in a cohort of young children affected by type II spinal muscular atrophy (SMA). Twenty-three children between 30 months and 6 years of age were treated with salbutamol (2 mg three times a day) for 1 year. All children were longitudinally assessed using the Hammersmith motor functional scale 6 months before treatment started (T0), at baseline (T1) and 6 and 12 months later. There was no significant change in function between T0 and T1 assessments, but the functional scores recorded after 6 and 12 months of treatment were significantly higher than those recorded at baseline (p = 0.006). Our results suggest that salbutamol may be beneficial to SMA patients without producing any major side effect. Larger prospective randomized, double-blind, placebo controlled trials are needed to confirm these preliminary findings. (c) 2008 Elsevier B.V. All rights reserved.

引用

页码：536 / 540

页数：5

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